Introduction to drug development

Question 1

What is the goal of the pre-discovery phase in drug development?

Answer 1

The goal of the pre-discovery phase is to find new information about disease processes, explore unexpected effects of existing treatments, and identify new technologies for more specific treatments.

Question 2

What is a drug target?

Answer 2

A drug target is a molecule, often a protein or enzyme, that the drug is designed to act upon in order to produce a therapeutic effect.

Question 3

What factors drive the discovery of new drugs?

Answer 3

Factors include medical need, disease prevalence, technical feasibility, research and development costs, and commercial considerations such as competition and market share.

Question 4

What are the two types of models used in preclinical studies?

Answer 4

The two types of models are in vitro (studying cells outside a living organism) and in vivo (studying within a living organism, such as mice or dogs).

Question 5

What are the main aims of preclinical studies?

Answer 5

The main aims are to determine the safety and efficacy of a drug on its target before proceeding to human trials.

Question 6

What is the purpose of a Phase 1 clinical trial?

Answer 6

Phase 1 trials aim to confirm the safety and tolerability of a drug by administering it to a small number of healthy volunteers.

Question 7

How does Phase 2 clinical trial differ from Phase 1?

Answer 7

Phase 2 trials test the new medicine on a small group of patients with the intended disease to evaluate efficacy, safety, and to identify the appropriate dose for further testing.

Question 8

What happens during a Phase 3 clinical trial?

Answer 8

Phase 3 trials involve a larger group of patients and compare the new medicine against existing treatments or a placebo to assess safety and efficacy in a real-world setting.

Question 9

What is marketing authorization in drug development?

Answer 9

Marketing authorization is the process of reviewing all data from preclinical and clinical trials to determine if a drug is safe and effective for its intended use in the studied population.

Question 10

What is post-market surveillance?

Answer 10

Post-market surveillance involves monitoring and collecting data on drugs after they have been approved to assess their real-world effectiveness and identify any long-term side effects.

Question 11

What are the different routes of administration for drugs?

Answer 11

Routes of administration include parenteral (injections), oral (tablets, capsules, liquids), and local (creams, patches, inhalation).

Question 12

What are some challenges in drug development?

Answer 12

Challenges include the lengthy and costly process, uncertainty about drug success, limitations of animal models, heterogeneity of patient populations, and increasing complexity of drug molecules.

Question 13

What is the average cost and length of time in developing a new drug?

Answer 13

The average cost of developing a new drug is approximately £2 billion. It takes 10 - 15 years to make a drug.

Question 14

Why has the number of new drugs approved declined?

Answer 14

The decline may be attributed to increased complexity in drug development, higher costs, and challenges in demonstrating efficacy and safety in diverse patient populations.

Question 15

What quality assurance programs are important in drug development?

Answer 15

Important quality assurance programs include Good Laboratory Practice (GLP), Good Manufacturing Practice (GMP), and Good Clinical Practice (GCP).

Question 16

What role do existing treatments play in the drug development pipeline?

Answer 16

Researchers explore existing treatments to identify unexpected effects or potential repurposing opportunities for new indications.

Question 17

How does technical feasibility influence drug target selection?

Answer 17

Technical feasibility ensures that the necessary expertise and technologies are available to effectively research and develop a drug targeting a specific molecule.

Question 18

What types of data are typically collected during preclinical studies?

Answer 18

Data collected during preclinical studies typically include safety profiles, pharmacokinetics, pharmacodynamics, and efficacy data using both in vitro and in vivo models.

Question 19

Why is patient population diversity a concern in clinical trials?

Answer 19

Patient population diversity is a concern because it affects the generalizability of trial results and may impact the drug’s effectiveness and safety across different demographics.

Question 20

What is the significance of escalating doses during Phase 1 trials?

Answer 20

Escalating doses during Phase 1 trials helps determine the maximum tolerated dose and safety profile, allowing researchers to identify the appropriate dosage for subsequent phases.

Question 21

How do researchers determine the correct dose for Phase 3 trials?

Answer 21

Researchers use data from Phase 2 trials, where various doses are tested, to identify the dose that maximizes efficacy while minimizing side effects for Phase 3 trials.

Question 22

What documentation is required for marketing authorization?

Answer 22

Documentation for marketing authorization includes comprehensive data from preclinical and clinical trials, proposed labelling, and patient information sheets.

Question 23

What are the main objectives of post-market surveillance?

Answer 23

The main objectives of post-market surveillance are to monitor long-term safety, assess real-world effectiveness, and identify any rare or unexpected side effects that may arise after approval.

Question 24

How can new technologies impact the drug development process?

Answer 24

New technologies can enhance drug targeting, improve formulations, streamline research processes, and facilitate more precise clinical trial designs.

Question 25

What are some examples of challenges posed by complex molecules in drug development?

Answer 25

Challenges include poor solubility, bioavailability issues, and difficulties in manufacturing and delivering complex biologics or synthetic drugs.